KR20250004924A - 메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산 - Google Patents

메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산 Download PDF

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KR20250004924A
KR20250004924A KR1020247040420A KR20247040420A KR20250004924A KR 20250004924 A KR20250004924 A KR 20250004924A KR 1020247040420 A KR1020247040420 A KR 1020247040420A KR 20247040420 A KR20247040420 A KR 20247040420A KR 20250004924 A KR20250004924 A KR 20250004924A
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adenine
nucleotides
poly
adenine nucleotides
tail
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크리스챤 돔브로브스키
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인텔리아 테라퓨틱스, 인크.
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    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07HSUGARS; DERIVATIVES THEREOF; NUCLEOSIDES; NUCLEOTIDES; NUCLEIC ACIDS
    • C07H21/00Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids
    • C07H21/02Compounds containing two or more mononucleotide units having separate phosphate or polyphosphate groups linked by saccharide radicals of nucleoside groups, e.g. nucleic acids with ribosyl as saccharide radical
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/11DNA or RNA fragments; Modified forms thereof; Non-coding nucleic acids having a biological activity
    • C12N15/113Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N15/00Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
    • C12N15/09Recombinant DNA-technology
    • C12N15/63Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/475Growth factors; Growth regulators
    • CCHEMISTRY; METALLURGY
    • C07ORGANIC CHEMISTRY
    • C07KPEPTIDES
    • C07K14/00Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/52Cytokines; Lymphokines; Interferons
    • C07K14/521Chemokines
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPR]
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
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    • C12N2830/00Vector systems having a special element relevant for transcription
    • C12N2830/50Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • CCHEMISTRY; METALLURGY
    • C12BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12NMICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/14Hydrolases (3)
    • C12N9/16Hydrolases (3) acting on ester bonds (3.1)
    • C12N9/22Ribonucleases [RNase]; Deoxyribonucleases [DNase]

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  • Genetics & Genomics (AREA)
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  • Organic Chemistry (AREA)
  • Molecular Biology (AREA)
  • Biotechnology (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Wood Science & Technology (AREA)
  • Zoology (AREA)
  • General Engineering & Computer Science (AREA)
  • Biochemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Microbiology (AREA)
  • Plant Pathology (AREA)
  • Biophysics (AREA)
  • Physics & Mathematics (AREA)
  • Medicinal Chemistry (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Saccharide Compounds (AREA)
  • Crystallography & Structural Chemistry (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)
  • Preparation Of Compounds By Using Micro-Organisms (AREA)
KR1020247040420A 2017-08-15 2018-08-14 메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산 Pending KR20250004924A (ko)

Applications Claiming Priority (4)

Application Number Priority Date Filing Date Title
US201762545883P 2017-08-15 2017-08-15
US62/545,883 2017-08-15
KR1020207007211A KR102740888B1 (ko) 2017-08-15 2018-08-14 메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산
PCT/US2018/046772 WO2019036513A1 (en) 2017-08-15 2018-08-14 STABILIZED NUCLEIC ACIDS ENCODING MESSENGER RIBONUCLEIC ACID (MRNA)

Related Parent Applications (1)

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KR1020207007211A Division KR102740888B1 (ko) 2017-08-15 2018-08-14 메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산

Publications (1)

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KR20250004924A true KR20250004924A (ko) 2025-01-08

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KR1020247040420A Pending KR20250004924A (ko) 2017-08-15 2018-08-14 메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산
KR1020207007211A Active KR102740888B1 (ko) 2017-08-15 2018-08-14 메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산

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KR1020207007211A Active KR102740888B1 (ko) 2017-08-15 2018-08-14 메신저 리보핵산(mRNA)을 암호화하는 안정화된 핵산

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US (3) US11673911B2 (https=)
EP (1) EP3668980A1 (https=)
JP (2) JP2020532961A (https=)
KR (2) KR20250004924A (https=)
CN (2) CN111212908B (https=)
AU (2) AU2018318135B2 (https=)
BR (1) BR112020003026A2 (https=)
CA (1) CA3072960A1 (https=)
CO (1) CO2020002989A2 (https=)
MX (1) MX2020001821A (https=)
WO (1) WO2019036513A1 (https=)

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ES3015369T3 (en) * 2014-10-09 2025-05-05 Seattle Childrens Hospital Dba Seattle Childrens Res Inst Long poly (a) plasmids and methods for introduction of long poly (a) sequences into the plasmid
AU2015330699B2 (en) 2014-10-10 2021-12-02 Editas Medicine, Inc. Compositions and methods for promoting homology directed repair
EP3555297A1 (en) 2016-12-19 2019-10-23 Editas Medicine, Inc. Assessing nuclease cleavage
JP7229923B2 (ja) 2017-01-06 2023-02-28 エディタス・メディシン、インコーポレイテッド ヌクレアーゼ切断を評価する方法
US11499151B2 (en) 2017-04-28 2022-11-15 Editas Medicine, Inc. Methods and systems for analyzing guide RNA molecules
MX2019014640A (es) 2017-06-09 2020-10-05 Editas Medicine Inc Nucleasas de repeticiones palíndromicas agrupadas y regularmente interespaciadas de proteina asociada 9 (cas9) genomanipuladas.
US11866726B2 (en) 2017-07-14 2024-01-09 Editas Medicine, Inc. Systems and methods for targeted integration and genome editing and detection thereof using integrated priming sites
CA3072960A1 (en) * 2017-08-15 2019-02-21 Intellia Therapeutics, Inc. Stabilized nucleic acids encoding messenger ribonucleic acid (mrna)
KR102832035B1 (ko) * 2017-09-29 2025-07-09 인텔리아 테라퓨틱스, 인크. 지질 나노파티클을 이용한 mRNA 전달의 체외 방법
EP3823633A4 (en) 2018-06-29 2023-05-03 Editas Medicine, Inc. SYNTHETIC LEAD MOLECULES, COMPOSITIONS AND METHODS RELATED THERETO
US12521451B2 (en) 2019-11-08 2026-01-13 Regeneron Pharmaceuticals, Inc. CRISPR and AAV strategies for x-linked juvenile retinoschisis therapy
EP4389901A4 (en) * 2021-08-17 2025-08-06 Sk Bioscience Co Ltd METHOD FOR STABLY MAINTAINING A POLY TAIL (A)
TW202325850A (zh) 2021-11-29 2023-07-01 大陸商上海瑞宏迪醫藥有限公司 Aadc、gdnf多核苷酸及其用於治療帕金森病
AU2023220237A1 (en) 2022-02-21 2024-08-15 Shanghai Regenelead Therapies Co., Ltd Vegf-binding molecule and pharmaceutical use thereof
CN117701602A (zh) * 2022-08-26 2024-03-15 深圳瑞吉生物科技有限公司 在宿主细胞中稳定存在的多段化PolyA多核苷酸及其应用
WO2024179544A1 (zh) * 2023-03-01 2024-09-06 仁景(苏州)生物科技有限公司 编码rna的工程化dna分子
TW202508610A (zh) 2023-08-23 2025-03-01 大陸商上海瑞宏迪醫藥有限公司 醫藥組成物及其用途
CN119799695A (zh) * 2023-10-10 2025-04-11 深圳瑞吉生物科技有限公司 提高mRNA稳定性以及蛋白表达的多元polyA多核苷酸及其应用
PL446809A1 (pl) * 2023-11-22 2025-05-26 Sieć Badawcza Łukasiewicz - PORT Polski Ośrodek Rozwoju Technologii Cząsteczki RNA ze zmodyfikowanymi sekwencjami końca 3' i ich zastosowanie
WO2025128871A2 (en) 2023-12-13 2025-06-19 Renagade Therapeutics Management Inc. Lipid nanoparticles comprising coding rna molecules for use in gene editing and as vaccines and therapeutic agents
GB202406051D0 (en) 2024-04-30 2024-06-12 Univ Oxford Innovation Ltd Segmented poly(a) tails
WO2026006390A2 (en) 2024-06-26 2026-01-02 Intellia Therapeutics, Inc. Modified guide rnas for genome editing
CN121628926A (zh) * 2024-08-29 2026-03-10 仁景(苏州)生物科技有限公司 一种多价流感mRNA疫苗

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Publication number Publication date
AU2018318135A1 (en) 2020-03-05
AU2018318135B2 (en) 2024-09-12
US12391722B2 (en) 2025-08-19
KR20200036025A (ko) 2020-04-06
US20250346625A1 (en) 2025-11-13
US20200172564A1 (en) 2020-06-04
WO2019036513A1 (en) 2019-02-21
EP3668980A1 (en) 2020-06-24
CO2020002989A2 (es) 2020-05-05
AU2024278094A1 (en) 2024-12-19
KR102740888B1 (ko) 2024-12-13
CA3072960A1 (en) 2019-02-21
US20230271997A1 (en) 2023-08-31
CN111212908A (zh) 2020-05-29
JP2020532961A (ja) 2020-11-19
MX2020001821A (es) 2020-09-18
CN111212908B (zh) 2025-01-24
BR112020003026A2 (pt) 2020-08-25
CN119899831A (zh) 2025-04-29
US11673911B2 (en) 2023-06-13
JP2023179428A (ja) 2023-12-19

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